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Spinal muscular atrophy (SMA) is a rare genetic disease that up until now has had no treatment for the underlying cause. While supportive care has been available, this is only able to minimise the disorder’s impact on patients’ quality of life and address complications that may arise. However, this has now changed thanks to a new drug.
Nusinersen - also called by its brand name Spinraza - is able to tackle the root cause of SMA by spurring the body on to produce survival motor neuron (SMN) protein. Individuals with the disease are lacking the gene known as SMN1, which usually fulfils this role. Without the vital protein, the body loses motor neurons in the spinal cord, meaning muscles cannot receive signals from the brain.
This leads to them wasting away, as well as causing muscle weakness, progressive loss of movement, and difficulty breathing and swallowing. Those with more severe forms of the disease often do not make it past the age of two, so a treatment that can help create more SMN proteins could be a huge breakthrough.
NICE had previously not been able to recommend nusinersen because of its high cost and uncertainties surrounding how effective the drug is in the long run. However, it has now reached an agreement with the drug’s manufacturer Biogen to make it available to NHS England.
At first, nusinersen will only be made available to the youngest patients diagnosed with SMA, as they tend to be the most severely affected. Older babies, children and young adults with SMA will be able to access it when it is rolled out following guidance from NICE being published in June.
Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said: “The committee has recognised that nusinersen is a promising treatment that has been shown to improve a range of outcomes important to patients. But it also recognised that there are significant uncertainties, particularly around its long-term benefits.
“All along we have felt it important to give all parties every opportunity to try to find a way to mitigate these uncertainties in order to make nusinersen available to patients in England.”
Written by James Puckle
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